The Changing Landscape of Opportunity for Cure of Severe Hemoglobinopathies in Middle-Income Regions

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Abstract
Thalassemia and sickle cell disease remain the most common life-threatening non-communicable diseases in children worldwide and an increasing burden on affected families and health services. Significant progress has been made in terms of technologies to improve access to a cure by both allogeneic and autologous gene-modified hematopoietic stem cell transplantation (HSCT). However, the high cost of cutting-edge treatments often places them beyond the reach of individual families or even national healthcare systems. Advances in frugal innovation and simplified HSCT procedures for low-risk transplants have significantly reduced the costs and complexities associated with HSCT without compromising on quality and outcomes. Because of the geographical distribution of hemoglobinopathies, i.e., largely in low- and middle-income countries (LMICs), HSCT cost optimization has the potential to impact a huge number of patients, increasing hope for a cure and health-related quality of life normalization, which in turn may affect supportive care compliance. Furthermore, because of the high burden of disease, LMIC transplant centers are rapidly increasing in number and developing unique expertise for the cure of thalassemia and sickle cell disease, particularly in India, where the Sankalp India Foundation with the support of DKMS and Cure2Children has implemented several cost-conscious transplant services. In fact, the very high success rate, increasing cost-effectiveness of transplantation, as well as the chronic nature of these conditions make them ideal initial candidates for start-up transplant centers, so it is likely that the global capacity for a cure for severe hemoglobinopathies will substantially increase in the years to come.

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